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Pediatrics. 2014 Jun;133(6):e1759-63. doi: 10.1542/peds.2013-1787.

Eculizumab as first-line therapy for atypical hemolytic uremic syndrome.

Author information

1
Klinik für Kinder- und Jugendmedizin, Pädiatrische Intensivstation, Goethe-Universität, Frankfurt/Main, Germany; martin.christmann@kispi.uzh.ch.
2
Clementine Kinderkrankenhaus, Pädiatrische Nephrologie, Frankfurt/Main, Germany; and.
3
Bioscientia Institut für Medizinische Diagnostik GmbH, Humangenetik, Ingelheim, Germany.
4
Klinik für Kinder- und Jugendmedizin, Pädiatrische Intensivstation, Goethe-Universität, Frankfurt/Main, Germany;

Abstract

Atypical hemolytic uremic syndrome (aHUS) is a genetic, life-threatening, chronic disease that can affect patients of all ages. aHUS is caused by uncontrolled complement activation due to genetic defects of complement regulation. Plasma exchange or infusion has been used to manage aHUS and may transiently maintain hematologic variables in some patients, but as the underlying complement dysregulation persists, end-stage renal disease or death occurs in 33% to 40% of patients during the first clinical manifestation. Here we present a pediatric case showing that first-line eculizumab treatment successfully blocked the progression of thrombotic microangiopathy in aHUS.

KEYWORDS:

atypical hemolytic uremic syndrome; complement; eculizumab; infant

PMID:
24843059
DOI:
10.1542/peds.2013-1787
[Indexed for MEDLINE]
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